Episode
Genetic Mechanic: Stanley Crooke on Fixing Nano-Rare Disease with Oligos
- Podcast
- Dose of Discovery
- Published
- May 27, 2026
- Duration seconds
- 3530
- Processing state
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- https://shows.acast.com/dose-of-discovery/episodes/genetic-mechanic-stanley-crooke-on-fixing-nano-rare-disease
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Summary
In this episode of Dose of Discovery , hosts Søren and AJ speak to physician-scientist Stanley Crooke, CEO of the nonprofit n-Lorem Foundation, about how antisense oligonucleotide (ASO) technology has evolved into a revolutionary therapeutic modality. Their conversation explores Stan's expansive career journey, from his early days building cancer programs at Bristol Labs and SmithKline to his bold decision to launch Ionis in 1989 when the industry doubted ASO technology. Stan discusses the emotional rollercoaster of biotech drug development, the inefficiencies of traditional small molecule discovery, and what led him to establish the n-Lorem Foundation. He shares how the regulatory frontiers of treating nano-rare genetic conditions “for free, for life” are changing rapidly and why innovations like patient-derived stem cells are driving drug development forward, one patient at a time. In this episode: 03:41 – Explainer: What are ASOs? 06:50 – Stanley Crooke’s journey and his journey into biomedicine 10:31 – Frustrations with big pharma, founding Ionis 15:05 – Trailblazing on ASO medicinal chemistry 19:33 – Building company culture amid clinical failure 22:56 – Spinraza (nusinersen) and the economics of drug pricing 28:08 – Launching the n-Lorem Foundation for nano-rare diseases 30:57 – “Free for life” ASO therapies 36:55 – Impact on n-Lorem’s patients 39:36 – “Everything we thought we knew about developmental delay is wrong.” 34:58 – Redefining traditional diseases n-of-1 trial success 26:12 – “Just tell me what the cause is. I'll fix it.” 37:22 – 90% success rate for n-of-1 work 45:08 – Moving beyond animal models with patient-derived iPSCs 52:23 – Biology is shifting industry toward platforms 55:55 – Wrap up and key takeaways About the guest Physician-scientist Stanle…